ProQR’s retinitis pigmentosa treatment gets orphan drug designation

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QR-1123, a first-in-class investigational antisense oligonucleotide for the treatment of vision loss associated with autosomal dominant retinitis pigmentosa, received an orphan drug designation from the FDA, ProQR announced in a press release.

“We are pleased to have orphan drug designation for our QR-1123 program targeting autosomal dominant retinitis pigmentosa, or adRP,” said Daniel de Boer, CEO of ProQR. “It highlights the unmet need for patients with this progressive disease-causing blindness. Our goal is to develop and actively advance a pipeline of programs that can treat inherited retinal diseases like adRP in a targeted manner.”

QR-1123, which is designed to be administered through intravitreal injections, aims to inhibit the toxic version of the rhodopsin protein by binding the P23H mutation in the RHO gene.

Discovered and developed by Ionis Pharmaceuticals, it was licensed by ProQR in 2018 and has since received IND clearance and a fast track designation from the FDA.